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We must unlock the full potential of cell and gene therapies

4 min read

In July, I was delighted to chair a Policy Connect roundtable on cell and gene therapies, to consider how they can contribute to better patient care, the wider health service and the economy.

A panel representing the NHS, academia, industry, and patients met to look at the challenges and opportunities these therapies present for manufacturing, research and the health service and the huge potential benefit for patients with a variety of currently incurable diseases. We drew out pointers for the regulatory environment, for skills and for patient pathways, and for how the UK can build on its real strengths in this area.

Cell and gene therapies are personalised medicines that involve extracting a patient’s own cells, fixing the disease-causing fault, and injecting them back into the patient. This process is simple for the patient and creates the potential for a permanent cure.

However, it is complex for researchers and manufacturers working to create personalised medicines at a scale that can gain regulatory approval and ultimately reach patients who need them. In part because of this complexity, cell and gene therapies are currently some of the most expensive products approved for use in the NHS.

However, they also represent the future of medicine. Their ability to provide a treatment at the source of disease means that they could be used to cure conditions as diverse as cancer, heart disease, arthritis or sight loss. These treatments could save lives and increase the quality of life for patients who currently may spend decades in chronic pain.

We need a regulatory environment that supports cell and gene as well as other personalised medicines

Sian Pursglove, the mother of a patient, described the difference in her daughter that this individually-tailored treatment had brought. Sian’s daughter had been on long-term treatments with a serious side effects when she was placed on a trial for cell and gene therapy. She transformed within weeks to a bright and happy little girl who could return to school during her treatment.

Cell and gene therapies offer hope for patients with a variety of diseases that are currently considered incurable. They can provide highly skilled jobs in manufacturing and clinical research. The UK is currently a world leader in their research and development. Centres across the country carry out around 12 per cent of all global clinical trials in this area, compared with just 3 per cent on average across healthcare. The UK has established a range of incentives to speed up research, production and access to market for these therapies and has established a strong and fast-growth sector.

As cell and gene therapies become more established, their price will fall, but this is not the only barrier to success. We need a manufacturing environment that encourages research and production in a global medicines market. We need development and delivery supply chains, and we need a clinical workforce with the resource and skill to deliver cell and gene therapy.

Embedding cell and gene therapy also requires a regulatory process that works for the new age of research and development, with personalised medicines increasingly at the forefront of health care.

It is often difficult to collect large scale clinical trial data on these treatments, and the cost-benefit assessment needs to consider the benefit to quality and quantity of life not just for the patient, but also their family and society at large.

This creates new regulatory challenges. The National Institute for Health and Care Excellence (NICE) has recently published a consultation on its ongoing methods review. It covers recommendations about how the cost and benefit of new technologies or medicines should be assessed as well as the measures that can be taken to reduce cost and increase accessibility.

In order to maintain the UK’s reputation as an attractive place to research, manufacture and deliver health innovations such as cell and gene therapies, we will need to go further. We need to develop a pathway from manufacture to patients that is clear and simple for industry, researchers, clinicians and patients alike. We need a regulatory environment that supports cell and gene as well as other personalised medicines, and we need the training and support for staff along the pathway to deliver them.

We have a unique opportunity as we recover from the Covid-19 pandemic to learn lessons and implement change. If we act proactively now, cell and gene work can contribute to a modern health service, with trusted research and innovation at its core, that will benefit patients now and for years to come.


Daniel Zeichner is the Labour MP for Cambridge and chair of the APPG for Data Analytics.

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